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An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051

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ClinicalTrials.gov Identifier: NCT03675126
Recruitment Status : Recruiting
First Posted : September 18, 2018
Last Update Posted : January 23, 2019
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics

Brief Summary:
The purpose of this extension study is to evaluate the safety, tolerability, and pharmacokinetics of repeat administrations of SRP-5051 in patients with Duchenne muscular dystrophy (DMD) who participated in studies of SRP-5051.

Condition or disease Intervention/treatment Phase
Muscular Dystrophy, Duchenne Drug: SRP-5051 Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051
Actual Study Start Date : December 19, 2018
Estimated Primary Completion Date : November 1, 2020
Estimated Study Completion Date : November 1, 2020


Arm Intervention/treatment
Experimental: SRP-5051
Participants to receive SRP-5051 via intravenous (IV) infusion. Dosage and frequency will be determined from the safety profile of other ongoing SRP-5051 (NCT03375255) study.
Drug: SRP-5051
SRP-5051 administered as an IV infusion.




Primary Outcome Measures :
  1. Number of participants with adverse events (AEs) [ Time Frame: From signing of informed consent to 4 weeks after the last infusion of SRP-5051 ]
    An AE is any untoward medical occurrence in a clinical trial participant, which does not necessarily have a causal relationship with the investigational drug. An AE can, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurs during or after administration of the study drug, whether or not considered related to the study drug.


Secondary Outcome Measures :
  1. Maximum plasma concentration (Cmax) of SRP-5051 [ Time Frame: End of infusion ]
    Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug infusion.

  2. Area under the plasma concentration versus time curve (AUC) of SRP-5051 [ Time Frame: Pre-dose, end of infusion, 6 hours post-dose ]
    Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug infusion.

  3. Number of participants with clinically relevant abnormalities, as assessed by vital sign measurements, physical examination findings, clinical laboratory tests and electrocardiograms (ECGs) [ Time Frame: From signing of informed consent to 4 weeks after the last infusion of SRP-5051 ]
    *A clinically relevant abnormality is an abnormality confirmed by repeat testing that is changed sufficiently from screening/baseline so that, in the judgment of the Investigator, a change in management is warranted.



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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

• Has completed Study 5051-101 (NCT03375255) and continues to meet the Safety Eligibility Criteria of Study 5051-101 (NCT03375255).

Exclusion Criteria:

  • Initiation or change of dosing (except for modifications to accommodate changes in weight) since entering Study 5051-101 (NCT03375255) and while participating in this study for any of the following: angiotensin converting enzyme (ACE) inhibitors, angiotensin receptor blocking agents (ARBs), beta-blockers, potassium and steroids*.
  • Requires antiarrhythmic and/or diuretic therapy for heart failure.
  • Use of any herbal medication/supplement containing aristolochic acid.
  • Treatment with any experimental therapy since entering original study or any experimental gene therapy for the treatment of DMD at any time.
  • Participation in an interventional clinical trial since completing original study.

Other inclusion/exclusion criteria apply.

* The dose of steroids must remain constant except for modifications to accommodate changes in weight.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03675126


Contacts
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Contact: Medical information +1 888 727 3782 clinicaltrials@sarepta.com

Locations
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United States, Florida
NW FL Clinical Research Group, LLC Recruiting
Gulf Breeze, Florida, United States, 32561
Contact: Shae Colbert    850-934-1299    scolbert@nwflcrg.com   
Principal Investigator: Weldon Mauney, MD         
United States, Georgia
Rare Disease Research, LLC Recruiting
Atlanta, Georgia, United States, 30318
Contact: Han C Phan, MD    678-883-6897    info@rarediseaseresearch.com   
Principal Investigator: Han C Phan, MD         
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Contact: Katherine Roath    913-945-9928    kroath@kumc.edu   
Principal Investigator: Jeffrey Statland, MD         
United States, Pennsylvania
Children's Hospital of Pittsburgh of UPMC Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Jennifer Monahan    412-692-5176    jennifer.monahan@chp.edu   
Principal Investigator: Hoda Abdel-Hamid, MD         
Sponsors and Collaborators
Sarepta Therapeutics
Investigators
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Study Director: Medical Director Sarepta Therapeutics, Inc.

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Responsible Party: Sarepta Therapeutics
ClinicalTrials.gov Identifier: NCT03675126     History of Changes
Other Study ID Numbers: 5051-102
First Posted: September 18, 2018    Key Record Dates
Last Update Posted: January 23, 2019
Last Verified: January 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Sarepta Therapeutics:
Duchenne muscular dystrophy
Exon Skipping
DMD
Exon 51
Ambulatory
Pediatric
Nonambulatory
Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO)
Duchenne

Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked